An early spinal muscular atrophy (SMA) diagnosis with the help of a newborn screening program, and treatment with disease-modifying…
Articles by Andrea Lobo
An 8-month-old girl with a clinical and genetic diagnosis of spinal muscular atrophy (SMA) type 1 saw her…
A young girl whose muscle weakness and respiratory distress in infancy were treated only as such was diagnosed as a…
Researchers in China report having developed a way to quickly and more easily screen for people who carry SMN1 gene…
Among women of reproductive age in a southern region of China, one in every 59 tested in a screening program…
The case of a 4.5-year-old girl with spinal muscular atrophy (SMA) type 2 who had acute pain…
Voyager Therapeutics is collaborating with Novartis to discover and develop next-generation gene therapies for conditions like…
Researchers in the U.S. report using a gene-editing method to alter the SMN2 gene sequence, leading to normal levels of…
Revvity has launched its EONIS Q system for faster and simplified newborn molecular screening of spinal muscular…
NIDO-361, Nido Biosciences’ experimental oral treatment for spinal and bulbar muscular atrophy (SBMA), was seen to be…